The US Food and Drug Administration (FDA) has granted orphan drug designation to GeNO for use of inhaled nitric oxide (NO) in treatment of persistant pulmonary hypertension of the newborn (PPHN). The orphan drug designation was granted ...
Tags: orphan drug designation, FDA, persistant pulmonary hypertension
Athersys has received FDA orphan drug designation for its proprietary cell therapy, MultiStem, to treat Hurler's syndrome, also known as mucopolysaccharidosis type I or MPS-I. The orphan drug designation provides substantial potential ...
Baxter International's Gammagard liquid 10% [Immune Globulin Infusion (Human)] has got approval from the US Food and Drug Administration (FDA) as a treatment for multifocal motor neuropathy (MMN). Baxter has been given Orphan Drug ...
Tags: Gammagard liquid, FDA, MMN patients
The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has recommended Orphan Drug Designation (ODD) for Erytech Pharma's investigational product Enhoxy. The drug, which is used to treat sickle cell ...
Tags: orphan drug designation, Erytech product, debilitating disease
Arno Therapeutics has received orphan-drug designation for AR-42 to treat neurofibromatosis type 2 (NF2) in the Europe. AR-42 is a broad-spectrum deacetylation inhibitor of both histone and non-histone proteins. The orally ...
Tags: Orphan-Drug designation, inhibitor, AR-42, clinical product
SK Biopharmaceuticals, a South Korean pharmaceutical company, has received orphan drug designation status for carisbamate for the management of patients with infantile spasms (IS). Carisbamate has previously been shown to have ...
REGENX BioSciences has received FDA orphan drug designation for treatment of familial hypercholesterolemia (HoFH) using NAV rAAV8 vectors. The REGENX program consists of a NAV rAAV8 vector expressing a normal copy of the human ...
Tags: orphan drug designation, familial hypercholesterolemia, clinical study